As reported here in 2017, a drug called RGX-314 is being tested as a one-time sub-retinal injection for wet age-related macular degeneration (wAMD). A preliminary study showed that patients with wAMD had long-term vision stabilization and anatomic improvement following a single treatment with the investigational gene therapy.
Now, in a subgroup of patients with the longest follow-up, three of six showed sustained benefit from a single injection of RGX-314 for more than 1.5 years. In another cohort with shorter follow-up, nine of 12 patients remained free of rescue medication for up to 6 months.
None of 42 patients treated in five dose cohorts had drug-related serious adverse events (SAEs). The results were reported at the American Academy of Ophthalmology meeting in October 2019.
Investigation of RGX-314 with a phase IIb trial is proceeding.